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Tests treatment safety and results for Congenital Myasthenic Syndrome
Official title A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
ClinicalTrials.gov ID: NCT06436742
What this study is testing
What is ARGX-119?
ARGX-119 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for congenital myasthenic syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function.
- Phase 1: an early, usually small safety study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- At least 18 years of age.
- Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS).
- Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) must have been receiving the medication for more than 3 months and...
You likely can't join if
- Diagnosis of CMS due to mutation of any gene other than DOK7.
- Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk...
- History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for more than 5 years. Adequately treated...
- Pregnant or lactating state or intention to become pregnant during the study.
The study team makes the final eligibility decision.
Where it's taking place
- Sacramento, California, United States
- Chicago, Illinois, United States
- Ottawa, Canada
- Marseille, France
- Paris, France
- Milan, Italy
- Valencia, Spain
- Belfast, United Kingdom
- Oxford, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Sacramento, California, United States; Chicago, Illinois, United States; Ottawa, Canada; Marseille, France; Paris, France; Milan, Italy and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.